Abstract

Introduction:

Pluripotent stem cell (PSC)–derived cell products hold great promise for future clinical use in a variety of indications such as type I diabetes, cardiomyopathies, macular dystrophies or Parkinson’s disease. Increasing regulatory requirements for such advanced-therapy medicinal products (ATMPs) imply the need for standardized reagents and highly reproducible production procedures. Automation of PSC expansion, differentiation, and potentially product optimization through cell sorting may contribute to successful and cost-effective innovative therapies. Using our versatile and integrated GMP-compliant cell processing platform CliniMACS Prodigy® we previously developed a cultivation and expansion workflow for iPS cells. Now we have implemented the differentiation of PSCs into mesencephalic dopaminergic (mesDA) progenitor cells on the device. Adapting this differentiation protocol from embryonic body–based1 to fully adherent cultivation2 enabled straightforward upscaling of a lab protocol to a medium-scale production process within the closed system. Additionally, we designed a concise marker panel (patent pending) for flow cytometry–based quality control (QC), i.e., characterization of the resulting mesDA progenitors.